Rare Disease Strategy Urgently Needed to Help 3 Million Canadians

OTTAWA, Nov. 9, 2015 /CNW/ - The majority of the 2.8 million Canadians with rare diseases experience extraordinary barriers getting an accurate diagnosis, finding a specialist, and accessing healthcare and treatment. Today, in Ottawa, in a panel discussion organized by the Economic Club of Canada, participants called for immediate implementation of Canada's Rare Disease Strategy that would provide Canadians with rare diseases the same quality of healthcare as those with more common conditions like cancer, heart disease and diabetes.

In May, on Parliament Hill, the Canadian Organization for Rare Disorders (CORD) announced the Strategy, calling on the federal and provincial governments to work collaboratively with the rare disease community to address these barriers. The Strategy also calls for national standards for newborn screening, centres of expertise, and dedicated funding for rare disease research.

"Thanks to the input of healthcare providers, patients, scientists, volunteer caregivers, pragmatic financiers and visionary leaders, we have crafted a Strategy that is both ambitious and achievable," said Durhane Wong-Rieger, President and CEO of CORD. "We are optimistic that this new federal government will seize upon the opportunity to activate a "ready-made" Strategy and will immediately and dramatically improve the lives of children and adults with rare diseases."

"Public health strategies for cancer, heart disease and diabetes implemented decades ago have resulted in significant improvements in the burden of illness, quality of life and survival," said Wong-Rieger. "National plans for rare diseases have already been adopted in more than 30 other countries and now is the time for Canada to do the same."

Speaking at the event, Jonathan Pitre, 15, from the Ottawa area, and who suffers from a genetic disease that causes the skin to constantly blister, shear and scar, said, "I am fighting pain and my own rare disease every day, and I'm determined to help other Canadians who also suffer from rare disorders. Letting all Canadians know about us and the need for research and to help people with rare diseases is my next battle."

Fred Horne, Alberta's health minister from 2011 to 2014 and Adjunct Professor at the University of Alberta School of Public Health, moderated today's event. "It's time for Canada to catch up with other countries and recognize that access to rare disease therapeutics is part of mainstream healthcare. Canada's Rare Disease Strategy is a plan that we can implement now to ensure that Canadians living with rare diseases have access to testing, diagnosis, medical experts and emerging treatments on par with high-performing healthcare systems in other countries."

Promoting research and translating its results into solutions that benefit patients are important elements of the Strategy. "Canada, capitalizing on strong genomic research and an impressive clinical expertise, leads globally in rare disease identification," said Dr. Alex MacKenzie, Principal Investigator, Children's Hospital of Eastern Ontario Research Institute and Professor, Faculty of Medicine, University of Ottawa. "We need to capitalize on the Strategy to both extend these discoveries to that large proportion of Canadians who are still without a diagnosis, as well as to formulate effective therapies for the even larger numbers who in 2015 have no treatment options."

"Canadian patients should be able to benefit from new treatments, however, this is not the reality for patients with rare diseases," said Sean Thompson, CEO, Ramsey Lake Pharma Corp., who provided a perspective from the pharmaceutical sector. "Entrepreneurs, scientists, governments and investors, must embrace a Rare Disease Strategy and commit to advancing and approving innovative treatments, enhancing access to breakthrough therapies, and ultimately, improving the lives of all patients with the added benefit of generating economic opportunities."

The Ottawa panel discussion is the third of five being held across the country by CORD and the Economic Club of Canada to discuss the need for prompt implementation of Canada's Rare Disease Strategy. Discussions have been held in Toronto and Edmonton, while others will follow in Montreal and Vancouver.

About Canada's Rare Disease Strategy

The Canadian Organization for Rare Disorders developed Canada's Rare Disease Strategy following extensive consultations with patients, caregivers, healthcare providers and researchers, as well as public and private-sector groups. Launched on Parliament Hill in May 2015, the Strategy establishes five major goals with a total of 20 related and practical actions. The five goals are: improving early detection and prevention; providing timely, equitable and evidence-informed care; enhancing community support; providing sustainable access to promising therapies; and promoting innovative research. For the complete plan, go to: www.raredisorders.ca/canadas-rare-disease-strategy/

About the Canadian Organization for Rare Disorders (CORD)

CORD is Canada's national network for organizations representing all those with rare disorders. CORD provides a strong common voice to advocate for health policy and a healthcare system that works for those with rare disorders. CORD works with governments, researchers, clinicians and industry to promote research, diagnosis, treatment and services for all rare disorders in Canada. www.raredisorders.ca

Follow the panel discussion and the Economic Club Series on Twitter: #Canada4Rare